Investing in Oncology: 4 Companies Driving Cancer Treatment Innovation

The oncology sector in the U.S. is on the verge of a transformative breakthrough. With the cancer therapeutics market already valued at over $170 billion in 2024 and expected to exceed $500 billion by 2034, the industry is experiencing a surge of innovation driven by cutting-edge treatments like immunotherapies and targeted therapies. As cancer rates continue to rise, particularly among children and underserved populations, the urgency for novel solutions has never been greater. Investors are increasingly drawn to this rapidly evolving field, where new therapies are not just improving survival rates but offering hope where there was none. With breakthroughs happening at an unprecedented pace, the oncology space presents an exciting and compelling opportunity for those looking to invest in the future of healthcare.

Now, let's take a look at a few promising stocks in this expanding sector that could be worth keeping an eye on.

OS Therapies (NYSE-A: OSTX) is a clinical-stage biotech company that's aiming to make a real difference in one of the toughest areas of medicine-osteosarcoma, a rare and aggressive bone cancer that mostly affects children and young adults. Their lead drug, OST-HER2, is not your typical cancer treatment. It's an off-the-shelf immunotherapy that uses a modified form of Listeria bacteria to kickstart the body's immune system into attacking cancer cells that express the HER2 protein.

So far, the results have been very promising. In a Phase 2b clinical trial involving 39 patients with recurrent osteosarcoma that had spread to the lungs but was surgically removed, OST-HER2 showed a statistically significant boost in 12-month event-free survival-33% compared to 20% in similar patients who didn't receive the treatment (p=0.0158). That's a big deal in a cancer type where survival rates haven't improved much in decades. "We believe OST-HER2 will make a significant difference in the treatment of osteosarcoma and welcome the opportunity to engage with FDA to get this investigational treatment to patients as quickly as possible," said Paul Romness, CEO of OS Therapies.

And the company is moving fast. OST-HER2 has already received special FDA and European designations, including:

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Rare Pediatric Disease Designation (RPDD)

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Fast Track Designation

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Orphan Drug Designation in both the U.S. and Europe

These help speed up the development and review process. OS Therapies is now preparing to submit a Biologics License Application (BLA) to the FDA by late 2025, with the hope of receiving Accelerated Approval shortly after.

In fact, the company recently requested a formal meeting with the FDA to agree on surrogate endpoints-essentially the key data the agency wants to see-in support of Breakthrough Therapy Designation and Accelerated Approval. That meeting is expected to happen in Q2 2025, and if all goes well, OST-HER2 could be approved by year-end. "We are excited to meet with the FDA... The goal is receiving Accelerated Approval for OST-HER2 by year-end 2025," said Dr. Robert Petit, OS Therapies' Chief Medical & Scientific Officer.

There's also potential upside beyond approval. If the drug is greenlit before September 30, 2026, OS Therapies is eligible for a Priority Review Voucher (PRV)-a valuable incentive from the FDA that the company could sell. A recent PRV sold for $150 million in February 2025, and that kind of windfall could help OS Therapies fund its next programs without needing to raise more money.

OST-HER2 isn't just being tested in people-it's also been conditionally approved by the USDA for treating osteosarcoma in dogs, which naturally develop the disease in much the same way as humans. Dogs with cancer have been shown to respond to OST-HER2, and researchers are using this data to better understand which biomarkers might predict a strong response in people.

Why does this matter? Because human and canine osteosarcoma share 96% genetic similarity. This growing field of Comparative Oncology is giving scientists like those at OS Therapies a unique window into how these therapies might work even before full human trials are complete. The treatment-and its story-will even be featured in the upcoming PBS documentary "Shelter Me: The Cancer Pioneers."

OST-HER2 is just the beginning. OS Therapies is also developing a next-generation Antibody Drug Conjugate (ADC) platform with tunable payloads-meaning they can design custom treatments for different cancers. This ADC program could offer future partnering or licensing opportunities, helping diversify the company's pipeline.

Financially, OS Therapies appears solid. The company completed both an IPO and private placement in 2024, raising $12 million and projecting enough cash to operate through mid-2026. With the heaviest clinical costs now behind them, OS Therapies expects lower spending moving forward. "We expect a significantly reduced outlay beginning in the second quarter of 2025," said Chris Acevedo, CFO of OS Therapies. "We have reduced our burn rate substantially... and expect cash on hand to last into 2026."

Investors interested in emerging biotech should keep a close eye on OS Therapies (OSTX). With:

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Strong Phase 2b results

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Imminent regulatory milestones

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High-value PRV opportunity

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A unique canine-human comparative oncology angle

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A growing immunotherapy and ADC pipeline

OSTX could be on the verge of something big. If OST-HER2 wins approval in late 2025, not only could it bring hope to children and families fighting this devastating disease, but it might also mark a major inflection point for the company's valuation and visibility in the biotech space.

Candel Therapeutics, Inc. (Nasdaq: CADL) is a clinical-stage biopharmaceutical company that focuses on developing off-the-shelf multimodal biological immunotherapies for cancer treatment. The company's approach includes two distinct immunotherapy platforms: one using genetically modified adenovirus (CAN-2409) and the other using herpes simplex virus (CAN-3110). CAN-2409 is currently being tested in phase 2a clinical trials for non-small cell lung cancer (NSCLC), and it has completed phase 2a and phase 3 trials in pancreatic cancer and localized prostate cancer, respectively. Meanwhile, CAN-3110, from the herpes simplex virus platform, is undergoing a phase 1b clinical trial in recurrent high-grade glioma (rHGG). Candel also employs its enLIGHTEN Discovery Platform to identify new viral immunotherapies for solid tumors.

In December 2024, Candel announced positive final survival data from a phase 2a clinical trial involving patients with borderline resectable pancreatic ductal adenocarcinoma (PDAC). Patients treated with CAN-2409 alongside standard care experienced a significant median overall survival benefit of 31.4 months, compared to just 12.5 months in the control group. The data indicated that long-term survivors in the treatment group had a substantially better survival rate, even in metastatic disease, highlighting the sustained benefits of CAN-2409. This promising data led the FDA to grant both fast-track and orphan drug designations for CAN-2409 in pancreatic cancer.

The company also revealed positive results from its phase 3 clinical trial of CAN-2409 in prostate cancer in December 2024. In this trial, CAN-2409, combined with standard radiation therapy, resulted in a 30% reduction in the risk of recurrence or death compared to the control group. In addition, the treatment arm demonstrated an 80.4% pathological complete response rate in post-treatment biopsies, compared to 63.6% in the control group. These findings are seen as a significant step toward regulatory approval for CAN-2409 in the treatment of localized prostate cancer, and the study was conducted under a Special Protocol Assessment (SPA) with the FDA.

Candel also provided an update on its phase 1b trial of CAN-3110 in recurrent high-grade glioma (rHGG). Data presented at the 6th Annual International Oncolytic Virotherapy Conference in October 2024 showed promising early survival outcomes, with three out of six patients surviving for over a year following repeated doses of CAN-3110. The company received both fast-track and orphan drug designations from the FDA for CAN-3110 in rHGG. Furthermore, Candel entered into a strategic partnership with IDEA Pharma, a biopharmaceutical strategy consultancy, in 2024. This collaboration will provide commercial insights into the development and commercialization of CAN-2409. Additionally, Dr. Elizabeth M. Jaffee, a leading pancreatic cancer expert, joined the company's Research Advisory Board, enhancing its focus on advancing treatments for pancreatic cancer.

In April 2025, Candel published the results of a phase 1b trial exploring the combination of CAN-2409 with nivolumab and standard care in newly diagnosed high-grade glioma patients. The data showed that the combination was well tolerated and resulted in extended survival for a subset of patients. A notable finding was the increase in T cell receptor (TCR) density and diversity, which correlated with improved survival outcomes. This reinforces the potential of CAN-2409 as a treatment for a wide range of solid tumors.

Financially, Candel reported research and development expenses of $4.8 million for the fourth quarter of 2024, down from $7.3 million the previous year. General and administrative expenses increased slightly to $3.3 million for the quarter. The company posted a net loss of $14.1 million for the quarter, compared to a net loss of $11.1 million in the same period the previous year. For the full year 2024, Candel's net loss amounted to $55.2 million, up from $37.9 million in 2023. As of December 31, 2024, the company had $102.7 million in cash and equivalents, which is expected to fund its operations into the first quarter of 2027.

Y-mAbs Therapeutics, Inc. (Nasdaq: YMAB) is a biopharmaceutical company that is making significant strides in the fight against pediatric cancer, particularly high-risk neuroblastoma. Neuroblastoma is a rare and aggressive cancer that mainly affects children, and Y-mAbs is dedicated to improving the outcomes for patients who are facing this challenging disease. The company has developed DANYELZA (naxitamab), the first FDA-approved treatment for patients with relapsed or refractory high-risk neuroblastoma in the bone or bone marrow. This breakthrough treatment offers hope to children who have exhausted other therapy options.

One of the most promising aspects of Y-mAbs is its work with naxitamab, a treatment that has shown strong efficacy in clinical trials. According to a Phase 2 clinical trial published in Nature Communications, naxitamab demonstrated a 50% overall response rate (ORR) among patients with relapsed or refractory high-risk neuroblastoma. Notably, the therapy showed particularly strong results in targeting residual disease in the bone and bone marrow, which are common areas where cancer cells hide and resist traditional chemotherapy.

Naxitamab is an anti-GD2 monoclonal antibody, meaning it targets the GD2 molecule found on the surface of neuroblastoma cells. This therapy is combined with granulocyte-macrophage colony-stimulating factor (GM-CSF), a substance that boosts the immune system to fight cancer. In clinical trials, naxitamab has shown manageable safety and promising efficacy, with many patients achieving a partial or complete response. In fact, 58% of patients with bone disease saw a positive response, making it an essential option for those who haven't responded to other treatments.

Y-mAbs has been actively working to expand the reach of DANYELZA globally. In 2024, the company reported significant revenue growth from its international markets, particularly in Europe and Asia. With DANYELZA now available in 69 U.S. centers and expanding into new regions, the company is well-positioned to bring this life-saving treatment to more children in need.

As of early 2025, Y-mAbs has adjusted its business strategy to focus on expanding the clinical development of its Radiopharmaceuticals Platform, alongside continuing to drive the growth of DANYELZA. The company has a robust pipeline, including its innovative SADA PRIT technology, which could open up new avenues for treating solid tumors in pediatric cancer patients.

Despite a net loss in 2024, Y-mAbs is focusing on cost efficiency and capital investment to fuel future growth. With a strong balance sheet and clear plans to expand its clinical and commercial efforts, Y-mAbs could offer significant upside potential for investors interested in the growing field of pediatric cancer therapeutics.

For investors looking to support innovative treatments in pediatric cancer, Y-mAbs Therapeutics presents a unique opportunity, with a solid product, promising clinical data, and an evolving strategy for growth.

Day One Biopharmaceuticals, Inc. (Nasdaq: DAWN) is making significant strides in pediatric cancer treatment, tackling one of the most critical areas of unmet need in the oncology space. The company's lead drug, OJEMDA (tovorafenib), is a promising targeted therapy designed to treat pediatric low-grade glioma (pLGG), a rare brain tumor that predominantly affects children.

OJEMDA is a Type II RAF kinase inhibitor, targeting BRAF mutations that are common in pLGG. Approved by the FDA under accelerated approval, the treatment is a breakthrough in a field where treatment options have been limited. Since its launch in April 2024, OJEMDA has shown strong sales, with net product revenues of $57.2 million for the full year. In the first eight months post-launch, over 1,600 prescriptions were written, highlighting the demand for new therapies in this space.

Day One has positioned OJEMDA as a cornerstone of its pipeline, and the company is already advancing its clinical strategy with a global, pivotal Phase 3 trial called FIREFLY-2. Full enrollment in the trial is expected by the first half of 2026, which could pave the way for further regulatory progress. Additionally, OJEMDA received Exclusively Pediatric designation from the Centers for Medicare & Medicaid Services in late 2024, which is expected to reduce the rebate percentage for the drug, further supporting its commercial viability.

The company's financial outlook appears solid, with a year-end 2024 cash balance of $531.7 million, providing a strong runway for future growth. Day One is also making progress in other areas, including its development of DAY301, a targeted antibody-drug conjugate (ADC), which has entered early-stage clinical trials. If successful, DAY301 could add to Day One's growing pipeline and diversify its offerings in the pediatric oncology market.

While Day One recorded a net loss in 2024, the company's focus on expanding access to OJEMDA and advancing its pipeline positions it well for long-term success. For investors, Day One represents a unique opportunity to support innovation in pediatric cancer therapeutics, with a promising product, a strong pipeline, and a dedicated team pushing the boundaries of what's possible in cancer treatment.

Disclaimers: RazorPitch Inc. "RazorPitch" is not operated by a licensed broker, a dealer, or a registered investment adviser. This content is for informational purposes only and is not intended to be investment advice. The Private Securities Litigation Reform Act of 1995 provides investors a safe harbor in regard to forward-looking statements. Any statements that express or involve discussions with respect to predictions, expectations, beliefs, plans, projections, objectives, goals, assumptions, or future events or performances are not statements of historical fact and may be forward-looking statements. Forward-looking statements are based on expectations, estimates, and projections at the time the statements are made that involve a number of risks and uncertainties that could cause actual results or events to differ materially from those presently anticipated. Forward-looking statements in this action may be identified through the use of words such as projects, foresee, expects, will, anticipates, estimates, believes, understands, or that by statements indicating certain actions & quote; may, could, or might occur. Understand there is no guarantee past performance will be indicative of future results. Investing in micro-cap and growth securities is highly speculative and carries an extremely high degree of risk. It is possible that an investor's investment may be lost or impaired due to the speculative nature of the companies profiled. RazorPitch has been retained and compensated by O S Therapies Inc to assist in the production and distribution of content related to OSTX. RazorPitch is responsible for the production and distribution of this content. It should be expressly understood that under no circumstances does any information published herein represent a recommendation to buy or sell a security. This content is for informational purposes only; you should not construe any such information or other material as legal, tax, investment, financial, or other advice. Nothing contained in this article constitutes a solicitation, recommendation, endorsement, or offer by RazorPitch or any third-party service provider to buy or sell any securities or other financial instruments. All content in this article is information of a general nature and does not address the circumstances of any particular individual or entity. Nothing in this article constitutes professional and/or financial advice, nor does any information in the article constitute a comprehensive or complete statement of the matters discussed or the law relating thereto. RazorPitch is not a fiduciary by virtue of any persons use of or access to this content.

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